Results

Alexion’s Soliris wows in rare disease PhIII trial, opening up a new franchise worth $500M-$700M

Three years after it launched, Alexion $ALXN says that the Phase III study of its cash cow Soliris for rare cases of relapsing neuromyelitis optica spectrum disorder scored a clear and convincing win, setting up some near term applications that one prominent analyst expects could be worth up to $700 million a year in new money.

Ludwig Hantson

The disease, NMOSD, is often confused with multiple sclerosis as the immune system attacks healthy tissue in the spine and eyes, responsible for blinding and sometimes killing patients. The drug inhibits the complement system, which investigators believe plays a key role in the autoimmune disorder.

The Phase III data were stellar. 

Investigators reported that the drug “reduced the risk of NMOSD relapse by 94.2% compared to placebo (p < 0.0001). At 48 weeks, 97.9% of patients receiving Soliris were free of relapse compared to 63.2% of patients receiving placebo.”

Geoffrey Porges, Leerink

Those numbers had Leerink’s Geoffrey Porges singing the drug’s praises Monday morning. He estimated that this indication alone could be worth an added $500 million to $700 million a year for the franchise, which is one of the most expensive drugs in the world. Added Porges:

Though Soliris had successful results in phase I, we believed a positive phase III outcome was not appreciated by consensus given significant trial design changes from phase I to phase III that added uncertainty about the phase III trial. We would expect regulatory filings to be completed by year-end and for the drug to be available at least in the US by next year.

Investors liked the tone today, bidding up shares by 8% in early trading.

Alexion, under new management after CEO Ludwig Hantson took the helm last year, will now swiftly spread out around the world to hunt up some key approvals in the US, Europe and Japan. Hantson has been hustling a Soliris successor, ALXN-1210, to the market as he angles to build a pipeline of new drugs. In the meantime, though, the biotech remains dependent on Soliris, with rivals hoping to snatch the crown away.

Michael Levy

“The primary goal in treating NMOSD is relapse prevention as each relapse further increases disability, which makes this disease so devastating. For decades, we have been hoping for a therapy that can prevent relapse and subsequent accumulation of disability by addressing a critical underlying cause of the disease,” said Michael Levy, associate professor at Johns Hopkins University.


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